GENETIC THERAPIES FOR HUNTINGTON'S DISEASE FAIL IN CLINICAL TRIALS

GENETIC THERAPIES FOR HUNTINGTON'S DISEASE FAIL IN CLINICAL TRIALS

Two pharmaceutical companies have halted clinical trials of gene-targeting therapies for Huntington's disease (HD), following the drugs' disappointing performance. Optimism around the Roche drug soared after the phase I/II trial showed that tominersen significantly lowered levels of mutant...

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Veröffentlicht in:Nature (London) 2021-05, Vol.593 (7858), p.180-180
1. Verfasser: Kwon, Diana
Format: Artikel
Sprache:eng
Schlagworte:
Amyotrophic lateral sclerosis
Cerebrospinal fluid
Clinical trials
Drug dosages
Huntingtin
Huntington's disease
Huntingtons disease
Life sciences
Mutation
Nucleotides
Optimism
Pharmaceutical industry
Proteins
Side effects
Single-nucleotide polymorphism
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Zusammenfassung:Two pharmaceutical companies have halted clinical trials of gene-targeting therapies for Huntington's disease (HD), following the drugs' disappointing performance. Optimism around the Roche drug soared after the phase I/II trial showed that tominersen significantly lowered levels of mutant huntingtin in the cerebrospinal fluid, without serious side effects. Guarded optimism Wave Life Sciences' trials were testing ASOs that leave the healthy version of huntingtin intact by targeting small mutations - known as single nucleotide polymorphisms (SNPs) - that occur only in the faulty gene.
ISSN:0028-0836
1476-4687
DOI:10.1038/d41586-021-01177-7